Pioneering clinical trials for children and young people with cancer
29 Oct 2026, 10 a.m.
We are pleased to announce over £7.5 million of funding for two new gene therapy clinical trials for children and young people with cancer.
For families at Great Ormond Street Hospital (GOSH) and others like them, research is often a critical source of hope.
At Great Ormond Street Hospital Charity (GOSH Charity), we are committed to funding research into better treatments for rare and life-limiting disease.
More research is needed into childhood cancer
Despite significant advances over the last few decades, cancer remains the biggest killer of children aged one to 14 in the UK*. Compared to adult cancers, research into children’s cancers has not progressed quickly enough and has been hampered by lower levels of funding.
While survival in some types of childhood cancers has improved, there has been little progress for the rarest and hardest to treat cancers. The mission of our cancer research strategy is to transform the outcomes and experiences for these children and young people. We are dedicating funding to the development of new treatments that are more effective and targeted, and that is what these clinical trials hope to deliver.
Two novel CAR T-cell therapy clinical trials
CAR T-cell therapy is a novel treatment that reprogrammes immune cells, called T-cells, to recognise and attack cancer.
GOSH researchers have led the way in using CAR T-cell therapy to treat childhood cancers. This pioneering approach has been very successful in acute lymphoblastic leukaemia (ALL). This work has led to a licensed CAR T-cell therapy on the NHS for children with relapsed ALL.
However, this success has been hard to replicate for other forms of childhood cancer.
These two newly funded clinical trials at GOSH are testing new treatment approaches designed to overcome the barriers that prevent CAR T-cell therapy from working in these cancer types.
First-in-human clinical trial for relapsed and refractory acute myeloid leukaemia in children and young people
Acute myeloid leukaemia (AML) is a form of cancer that affects blood-producing cells in the bone marrow. Treatment includes chemotherapy to destroy the leukaemia cells and sometimes a stem cell transplant to replace the cells lost with healthy blood-producing cells.
For children and young people who don’t respond to treatment or whose leukaemia comes back, their options are very limited.
This funding from GOSH Charity will enable the team, led by Professor Persis Amrolia, to run a clinical trial for children and young people whose AML has come back or is resistant to treatment.
To date, CAR T-cell treatment for AML has been challenging because the targets on leukaemia cells are also found on healthy stem cells. In this study, for the first time, Prof Amrolia’s team will genetically edit donor stem cells to make them invisible to the CAR T-cells before transplanting into the patient. This will allow the CAR T-cells to fight the leukaemia whilst sparing healthy donor stem cells.
As well as potentially providing a novel treatment option for AML, this clinical trial also has wider potential as a proof-of-concept for combining gene-edited stem cell approaches with CAR T-cell therapies. If successful, this could offer new hope for children and young people with hard-to-treat AML. In the future, it could also help treat adults with this disease.
“I’m so grateful to GOSH Charity for funding this ground-breaking new study which may offer new hope for children with AML who have relapsed after conventional treatment”
Next-generation CAR T-cell therapy for children and young people with relapsed sarcoma
Sarcoma is type of cancer that develops in bone, muscle and other connective tissues. Childhood sarcomas are rare, and initial treatment involves a combination of chemotherapy, surgery and radiotherapy.
However, around one in three children with sarcoma either do not respond to treatment or the cancer returns after treatment.** For these children and young people, treatment options remain limited.
New funding from GOSH Charity will expand an ongoing clinical trial, led by Dr Karin Straathof, to test next-generation CAR T-cell therapy for children and young people with sarcoma that has come back or stopped responding to standard treatment.
By testing these improvements within a single clinical trial, the team hopes to speed up the development of a more effective treatment. Ultimately, this research aims to improve outcomes for children and young people with hard-to-treat sarcoma.
“In this clinical trial we will test different versions of next-generation CAR T cells to find most effective approach for patients with hard-to-treat sarcoma. We are very grateful for this funding.”
These trials are not yet open for patient recruitment. Any patients eligible to receive treatment under the NHS and interested in either of these trials should approach their specialist healthcare provider.
** The REFoRMS-SR project. Int J Cancer. 2024.